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Neuroscience at UMass Medical School.

Research topics on Neurology, Neurobiology, and Neuroscience at UMMS includes: 

Psychiatry and Psychiatric disorders, Alzheimer's diseasesAmyolateral Sclerosis (ALS), Opioid withdraw, and more. Browse our innovation by scrolling though below. 







Browse Our Inventions:



Title: AAV2-mediated Gene Delivery of sFasL as a Neuroprotective Therapy in Glaucoma. UMMS16-64; Patent Pending.

  • New AAV therapy facilitates long-term production of sFasLin the retina by intra-vitreal injection with no detrimental effect observed in normal animals. Glaucoma-prone mice were injected either before or after disease onset and both animals were found to have preserved retinal ganglion cells with their axons, which normally results in death with onset of the disease.

Title: Branched Oligonucleotides as Therapeutics. UMMS16-45; Patent Pending. 

  • This technology is based on the discovery that branched oligonucleotide structure improves the level of tissue retention in brain by more than 100 fold compared to non-branched compounds of identical chemical composition. The invention discloses branched oligonucleotides, specifically di-branched assymetric fully modified siRNAs, exhibiting great uniform distribution throughout the CNS and other target tissues, enhanced cellular uptake, minimal immune response and off-target effects, without formulation.

Title: Method to Enhance the Efficiency of Systemic AAV Gene Delivery to the Central Nervous System. UMMS16-19; Patent Pending.

  • This invention relates to compositions and methods for increased efficiency of gene transfer to the CNS. The invention is based on the surprising discovery that viral vector-mediated delivery of nucleic acids to the CNS of a subject can be enhanced by administering the viral vector (rAVV) with a composition comprising a highly hydrophilic molecule conjugated to a blood brain barrier (BBB)-receptor ligand (e.g., K16ApoE).

Title: Gene Therapy and/or Small Molecule Treatment to Correct Beta-Oxidation and Glycolysis in Neurodegenerative Disorders Involving N-Acetylaspartate (NAA) Metabolism and its Associated Metabolic Pathways. UMMS16-17; Patent Pending.

  • Newly discovered phenomenon in which changes in brain energy metabolism is observed when N-Acetylaspartate (NAA) levels are perturbed. Altered NAA levels are associated with Cavagan and neurodegenerative diseases where there is increased preference for utilization of fatty acids over glucose, leading to white matter loss.

Title: Gene Therapy and/or Small Molecule Treatment to Correct Beta-Oxidation and Glycolysis in Neurodegenerative Disorders Involving N-Acetylaspartate (NAA) Metabolism and its Associated Metabolic Pathways. UMMS16-17; Patent Pending.

  • Newly discovered relationship between N-Acetylaspartate (NAA) metabolite levels and glucose metabolism. Introduction of ASPA gene can reverse metabolic changes that occur with decreased levels of NAA. Monitoring and adjusting the NAA levels may have implication for wide range of diseases that display changes in bioenergetics metabolism.  

Title: Identification of Epigenetic Modifiers of the Silenced FMR1 Gene: Potential Targets for Fragile X Syndrome Therapeutics. UMMS16-03; Patent Pending. 

  • This invention comprises methods for treating Fragile X Syndrome and other FMR1-inactivation-associated disorders by inhibiting epigenetic silencers of the FMR1 gene. The invention discloses a variety of FMR1 modulators, functioning either through RNA interference or small molecule inhibition, for optimal FMR1 reactivation.

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Title: A Method to Quantify Net Axonal Transport in Motor Nerves. UMMS15-65; Patent Pending.  

  • This invention provides methods of diagnosing motor neuron pathologies either before or after treatment by administering to the subject a radiolabeled agent comprising tetanus toxic C fragment and assessing both transport across the neuromuscular junction and retrograde transport. This novel biomarker system can greatly facilitate the study and treatment of diseases such as ALS.

Title: Recombinant Glut1 Adeno-associated Viral Vector Constructs and Related Methods for Restoring Glut1 Expression. UMMS15-61; Patent Pending. 

  • This new rAAV gene therapy introduces Glut1, a primary glucose transporter in the blood brain barrier (BBB), to the brain through endothelial cells lining the brain vasculature. Glut1-deficiency syndrome is an autosomal-dominant disorder, a rare but debilitating childhood neurological disorder. Introducing Glut1 using this technology was shown to improve symptoms associated with Glut1 deficiency in mice, demonstrating potential use in patients with Glut1-deficiency syndrome. 

Title: CARDS: CRISPR Arrayed Repeat Detection System / Part I. UMMS15-58; Patent Pending. 

  • This invention discloses a platform to detect DNA repeat expansions called CRISPR Arrayed Repeat Detection System (CARDS). Utilizing this platform primary cell cultures and/or blood cell smears can be tested under conventional clinical diagnostic laboratory conditions to diagnose genetically-based diseases having DNA repeat expansions, including but not limited to ALS. Further disclosed are dCas9 constructs having fluorescent proteins bound to any or all stem loop sequences, wherein detection of a plurality of dCas9 constructs having different colored fluorescent proteins can simultaneously detect at least six (6) different gene target loci. 

Title: New XCI Inhibitors as Potential Rett Syndrome Therapeutics. UMMS15-53; Patent Pending.

  • This invention provides methods of treating a subject having a dominant X-linked disease, the method comprising administering to the subject an X chromosome inactivation factor (XCIF) inhibitor in an amount effective for inducing expression a target X-linked gene. The invention provides small molecule and oligonucleotide XCIF inhibitors. In some embodiments, the X-linked gene is MECP2 and the X-linked disease is Rett Syndrome. 

Title: Methods and System for Optimizing a Control System. UMMS15-40. Patent Pending. 

  • Inducing a switch in neuronal state using energy optimal stimuli is relevant to a variety of problems in neuroscience. Analytical techniques from optimal control theory can identify such stimuli; however, solutions to the optimization problem using indirect variational approaches can be elusive in models that describe neuronal behavior. Here we develop and apply a direct gradient-based optimization algorithm to find stimulus waveforms that elicit a change in neuronal state while minimizing energy usage. Our gradient-based algorithm: 1) enables automated exploration of a wide solution space, using stochastically generated initial waveforms that converge to multiple locally optimal solutions; and 2) finds optimal stimulus waveforms that achieve a physiological outcome condition, without a priori knowledge of the optimal terminal condition of all state variables. Analysis of biological systems using stochastically-seeded gradient methods can reveal salient dynamical mechanisms underlying the optimal control of system behavior. The gradient algorithm may also have practical applications in future work, for example, finding energy optimal waveforms for therapeutic neural stimulation that minimizes power usage and diminishes off-target effects and damage to neighboring tissue. 

Title: Ventricular Shunt Catheter Assembly. UMMS15-36; Patent Pending. 

  • The invention discloses a novel ventricular shunt designed to minimize damage during shunt revision surgery. The novel shunt has an outer catheter sheath that is placed in the first surgery and does not need to be removed even if catheter replacement is needed due to obstruction. It stays as a permanent catheter sheath and creates a permanent access tunnel into the brain ventricle.

Title: New AAV Vectors for Safe and Efficient Expression of Lysosomal Enzymes in the CNS. UMMS15-19; Patent Pending. 

  • New AAV vectors that enable transgene expression at therapeutic levels for treatment of lysosomal and other disorders. These levels are achieved without the adverse events that arises due to secondary effects of AAV-mediated product delivery. This newly engineered regulatory element can apply broadly to circumvent adverse effects of AAV therapy. 

Title: Hedgehog Signaling in Bipolar Affective Disorder. UMMS15-02; Patent Pending. Related Publication

  • The invention encompasses methods of treating psychiatric affective disorders by administering to the subject a therapeutically effective amount of an antagonist to Smoothened (Smo), an antagonist to Patched-1 (Ptch-1) and/or an antagonist to Sonic Hedgehog. The invention further provided methods for diagnosing affective disorders.

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Title: Novel High Efficiency Library-identified CNS-tropic AAV Vectors. UMMS14-59; Patent Pending. 

  • Newly designed recombinant AAV capsids with greater efficiency for CNS delivery than AAV9, the natural variant with the currently highest known CNS transduction efficiency. These new AAV capsids, B1-B4, show considerably lower off-target effects in the liver when compared to AAV9. Additionally, each of these AAV capsids show selectively higher efficiency for specific peripheral tissues (e.g. pancreas, sk. muscle, heart, adipocyte). 

Title: Novel High Efficiency Peptide Grafted CNS-tropic AAV Vectors. UMMS14-58; Patent Pending. 

  • New AAV technology with an insertion of 19-amino acid “alanine string” that has dramatically increased CNS tropism compared to that of AAV9, the natural variant with the currently highest known CNS transduction efficiency. This technology shows increased CNS transduction without changing transduction efficiency of peripheral tissues. 

Title: Modified Mullerian Inhibiting Substance (MIS) Proteins and Uses Thereof for the Treatment of Diseases. UMMS14-56; Patent Pending.

  • A new recombinant human MIS protein with improved effectivity may improve the treatment of cancer or neurodegenerative disease. This technology maximizes bioactivity using a leader sequence and a modified cleavage site that promotes increased product yield and MIS processing. This recombinant MIS protein also includes a tag to facilitate purification for research and therapeutic purposes. 


  • This new invention spans from the discovery of potent silencing site that was previously unidentified on Huntington gene (htt) through screening of more than 200 compounds. Conventional siRNAs will target this 3’ UTR site for potent inhibition.  Efficacy was demonstrated using different siRNA configurations in several cell types and primary neurons that may aid research and future therapeutic applications. 

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Title: MicroRNA Mediated Knockdown of SOD1 Using Recombinant Adeno-associated Vectors. UMMS13-19; Patent Pending. 

  • This invention discloses a novel miRNA delivered by rAAV for silencing SOD1 & C9orf72 SODl genes, which are associated with ALS. This method enables effective therapy at low doses with the persistence of rAAV episomes that continually expresses the nucleic acids, thus rendering re-treatment unnecessary. This method also minimizes rAAV exposure to non-CNS peripheral tissue.


  • This invention is directed to a diagnostic test for identifying individuals with amyotrophic lateral sclerosis (ALS) or individuals who are at risk of developing ALS. This test is based on detecting one or more alterations in a profilin 1 (PFN1) sequence of an individual. 

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  • This technology provides vectors that increase survival of motor neuron (SMN) protein production by an SMN2 gene and methods for treatment of spinal muscular atrophy (SMA). Further discloses is a clonal second generation SMN-luciferase reporter cell line that combines the strengths of both the promoter-based assay and a previous splicing reporter. This assay is much more robust, has lower well-to-well variation, and displays more stable luciferase expression that does not change with serial passage that can be used to identify potential therapeutic agents for SMA. 

Title: Rabbit & Guinea Pig Polyclonal Antibodies to the Circadian Proteins; NPAS2/MOP4, BMAL1/Arnt3/Arnt1/Mop3, Per2, CLOCK, PER1, Casein Kinase 1 delta, and Casein Kinase 1 epsilon. UMMS12-49.

  • Rabbit and Guinea Pig polyclonal antibodies to circadian protein available for licensing.
    1. anti-NPAS2: antigen is amino acids 597-916
    2. anti-BMAL1: antigen is amino acids 382-586
    3. anti-PER2: antigen is amino acids 1-200
    4. anti-CLOCK: antigen is amino acids 363-855
    5. anti-PER1: antigen is amino acids 1-23
    6. anti-CASEIN KINASE 1 DELTA: antigen is amino acids 243-415
    7. anti-CASEIN KINASE 1 EPSILON: antigen is residues 202-416


  • To better understand the risks associated with stem cell therapies, this invention provides compounds to track transplanted stem cells in vivo overtime using noninvasive imaging techniques.  Specifically, the invention provides novel multi-modality probes constructed by utilizing (a) the high selectivity of long hydrocarbon chains for binding to plasma membranes of cells, (b) a near-infrared (NIR) dye for optical imaging, and (c) a radionuclide for PET or SPECT imaging.

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  • This novel patented technology provides methods of treating peripheral neuropathy, brain injury, and neurodegenerative disease utilizing an siRNA that inhibits sterile a/ Armadillo/To 11-Interleukin receptor homology domain protein (SARM) expression. This invention is based on the discovery that inhibition of SARM mRNA reduces axonal and synaptic degeneration in mice. The invention further provides methods of identifying potential therapeutic agents that modulate SARM. 

Title: Modulation of Ubiquitination of Synaptic Proteins for the Treatment of Neurodegenerative and Psychiatric Disorders. UMMS11-03; Patent Pending. 

  • This invention is a potential therapeutic for neurological and psychiatric disorders in which increased cell surface expression of AMPA, NMDA, and D1 dopamine receptors is desired. This technology is based on the discovery that inhibition of PSD-95, Mdm2, or Mdm4 decreases endocytosis of glutamate, NMDA, and dopamine receptors thereby modulating synaptic plasticity. The invention further provides screens for additional therapeutic agents targeting Mdm2, Mdm4, and PSD-95. 

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Title: ANTI-SOD1 ANTIBODIES AND USES THEREOF. UMMS10-69; Patent 9,109,037.

  • This invention discloses a panel of newly discovered human monoclonal antibodies against human SOD1 protein.  There are two types of SOD1 antibodies available: 1) one that binds to all forms of SOD1 and 2) one that binds to the misfolded form of SOD1. Mutated SOD1 is associated with 20% of inheritable forms of Amyotrophic Lateral Sclerosis (ALS) and blocking its function is protective. These monoclonal antibodies present a unique therapeutic opportunity to treat ALS with an immunologically targeted approach. 


  • New effective and safe gene therapy approach that allows gene delivery across the blood brain barrier (BBB) upon intravascular administration. Designed to minimize off-target effects by incorporation of non-CNS-tissue specific miRNA binding site into the transgene expression cassette. 


  • AAV10 vector-based gene therapy for treating ALS with enhanced tropism for neuronal cells. Administration of rAAV encoding inhibitory RNA for superoxide dismutase 1 (SOD1) distributes widely throughout CNS with low toxicity. Long-term inhibition of mutant SOD1 improves lifespan in the animal models. 

Title: Immunotherapy for T Cell-Mediated Autoimmune Diseases Using ITK Inhibitors. UMMS10-05; Patent Pending. 

  • The present disclosure is based, in part, on the discovery that interleukin-2-inducible T cell kinase (ITK) and CD28 signals regulate auto-reactive T cell trafficking and that organ-specific T cell mediated autoimmune diseases such as Type 1 diabetes and multiple sclerosis. Accordingly, the present specification provides methods of treating organ-specific T cell mediated autoimmune diseases by inhibiting ITK with a therapeutically effective amount of an ITK inhibitor, e.g., BMS509744, ibrutinib, 10n, 2-amino-5-(thioaryl)thiazole, 5 aminomethylbenzimidazole, 2-amino-5-[(thiomethyparyl]thiazole, and biaryl thiophene. 

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Title: Mammalian Cell Lines for High Throughput Screening for mRNA Splicing Inhibitors. UMMS09-06.

  • This invention contains a method for high throughput screening for drug compounds that inhibit mRNA splicing. Using a luciferase based assay system, the inventors developed an assay that can report when splicing has occurred. Drugs that can inhibit major and minor splicesomes can be identified through this method. This technique is applicable to diseases that arise from perturbations in mRNA processing and/or splicing.

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Title: Kratom and its Alkaloidal Components as Therapy for Withdrawal from Prescription Opioid Pharmaceuticals. UMMS08-53; Patent Pending.  

  • This invention utilizes natural products derived from Kratom, a medicinal herb native to Thailand, to treat opioid withdrawal from prescription opioid agents. Mitragynine is the predominant alkaloid isolated from Kratom and has been found to bind to opioid receptors. This finding suggests a mechanism by which Kratom and its derived molecules may reduce opioid craving. These findings introduce a novel potential for the development of a naturally derived agent for the treatment and management of opioid addiction and withdrawal. 

Title: Use of Cathepsin B Inhibitors for the Treatment of IL-1 Related Diseases. UMMS08-51; Patent Pending.  

  • This innovation describes detailed mechanisms of how immune cells recognize microorganisms (non-self) from host (self). The cytoplasmic receptor complex NALP3 inflammasome has been found to react to a variety of crystals such as silica crystals or cholesterol crystals, all of which were found to require phagocytosis for activation. By inhibiting phagosomal acidification, the inventors were able to prevent activation of NALP3 in the presence of the crystal activators. Understanding these mechanisms may be valuable to research into IL-1 related diseases of sterile inflammation including atherlosclerosis, amyloidosis, Alzheimer silicosis, asbestosis and others. 

Title: Rabbit Polyclonal Antibody to mPer1. UMMS08-33.

Title: Rabbit Polyclonal Antibody to Per2. UMMS08-32.

Title: Guinea Pig Polyclonal Antibody to BMAL. UMMS08-31.

Title: Rabbit Polyclonal Antibody to CLOCK. UMMS08-30.